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Use of Defibrotide to help prevent post-transplant endothelial injury in a genetically predisposed infant with metachromatic leukodystrophy undergoing hematopoietic stem cell gene therapy.

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第一作者: Valeria,Calbi
第一单位: San Raffaele Telethon Institute for Gene Therapy (SR-TIGET), IRCCS San Raffaele Scientific Institute, Milan, Italy.;Pediatric Immunohematology Unit and Stem Cell Program, IRCCS San Raffaele Scientific Institute, Milan, Italy.
作者单位: San Raffaele Telethon Institute for Gene Therapy (SR-TIGET), IRCCS San Raffaele Scientific Institute, Milan, Italy.;Pediatric Immunohematology Unit and Stem Cell Program, IRCCS San Raffaele Scientific Institute, Milan, Italy. [1] San Raffaele Telethon Institute for Gene Therapy (SR-TIGET), IRCCS San Raffaele Scientific Institute, Milan, Italy.;Pediatric Immunohematology Unit and Stem Cell Program, IRCCS San Raffaele Scientific Institute, Milan, Italy.;Neurology Department, IRCCS San Raffaele Scientific Institute, Milan, Italy. [2] Pediatric Immunohematology Unit and Stem Cell Program, IRCCS San Raffaele Scientific Institute, Milan, Italy.;Vita-Salute San Raffaele University, Milan, Italy. [3] Pediatric Immunohematology Unit and Stem Cell Program, IRCCS San Raffaele Scientific Institute, Milan, Italy. [4] San Raffaele Telethon Institute for Gene Therapy (SR-TIGET), IRCCS San Raffaele Scientific Institute, Milan, Italy. [5] San Raffaele Telethon Institute for Gene Therapy (SR-TIGET), IRCCS San Raffaele Scientific Institute, Milan, Italy.;Pediatric Immunohematology Unit and Stem Cell Program, IRCCS San Raffaele Scientific Institute, Milan, Italy.;Vita-Salute San Raffaele University, Milan, Italy. [6] Pediatric Immunohematology Unit and Stem Cell Program, IRCCS San Raffaele Scientific Institute, Milan, Italy.;Unit of Hematology and Bone Marrow Transplantation, IRCCS San Raffaele Scientific Institute, Milan, Italy. [7] Department of Anesthesia and Critical Care, IRCCS San Raffaele Scientific Institute, Milan, Italy. [8] Pediatric Intensive Care Unit, Department of Anesthesia and Critical Care, Fondazione IRCCS Ca' Granda, Ospedale Maggiore Policlinico, Milan, Italy. [9] Bone Marrow Transplantation Unit, Great Ormond Street Hospital, London, UK. [10] Department of Paediatrics, Norfolk and Norwich University Hospital, Norwich, UK. [11] Neuroradiology Unit, IRCCS San Raffaele Scientific Institute, Milan, Italy. [12] Department of Chemistry, Biology and Biotechnologies, University of Perugia, Perugia, Italy. [13] HUS Center, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, Milan, Italy. [14] Neurology Department, IRCCS San Raffaele Scientific Institute, Milan, Italy. [15] San Raffaele Telethon Institute for Gene Therapy (SR-TIGET), IRCCS San Raffaele Scientific Institute, Milan, Italy.;Vita-Salute San Raffaele University, Milan, Italy. [16] Vita-Salute San Raffaele University, Milan, Italy.;Unit of Hematology and Bone Marrow Transplantation, IRCCS San Raffaele Scientific Institute, Milan, Italy. [17] San Raffaele Telethon Institute for Gene Therapy (SR-TIGET), IRCCS San Raffaele Scientific Institute, Milan, Italy. aiuti.alessandro@hsr.it.;Pediatric Immunohematology Unit and Stem Cell Program, IRCCS San Raffaele Scientific Institute, Milan, Italy. aiuti.alessandro@hsr.it.;Vita-Salute San Raffaele University, Milan, Italy. aiuti.alessandro@hsr.it. [18]
DOI 10.1038/s41409-017-0085-1
PMID 29379168
发布时间 2019-07-15
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